National Cancer Institute Thesaurus

Last uploaded: February 23, 2024
Preferred Name

Eliglustat

Synonyms

N-((1R,2R)-2-(2,3-Dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(pyrrolidin-1-ylmethyl)ethyl)octanamide

ELIGLUSTAT

Eliglustat

GENZ 99067

GENZ-99067

Definitions

A ceramide analog and an orally bioavailable inhibitor of glucosylceramide synthase (GCS; ceramide glucosyltransferase), that may be used to decrease the production of glycosphingolipids (GSLs) including glucosylceramide, also known as glucocerebroside. Upon oral administration, eliglustat inhibits GCS, which catalyzes the initial step in the synthesis of glucosylceramide and other GSLs. This decreases the production of glucosylceramide and other GSLs, which have important roles in various diseases and cellular processes, including immune processes and functions. Eliglustat may be used in substrate reduction therapy in diseases in which the enzyme glucocerebrosidase (GCase), responsible for the breakdown of glucocerebroside, is deficient.

ID

http://ncicb.nci.nih.gov/xml/owl/EVS/Thesaurus.owl#C96282

CAS_Registry

491833-29-5

Chemical_Formula

C23H36N2O4

code

C96282

Concept_In_Subset

http://ncicb.nci.nih.gov/xml/owl/EVS/Thesaurus.owl#C176424

http://ncicb.nci.nih.gov/xml/owl/EVS/Thesaurus.owl#C63923

Contributing_Source

FDA

DEFINITION

A ceramide analog and an orally bioavailable inhibitor of glucosylceramide synthase (GCS; ceramide glucosyltransferase), that may be used to decrease the production of glycosphingolipids (GSLs) including glucosylceramide, also known as glucocerebroside. Upon oral administration, eliglustat inhibits GCS, which catalyzes the initial step in the synthesis of glucosylceramide and other GSLs. This decreases the production of glucosylceramide and other GSLs, which have important roles in various diseases and cellular processes, including immune processes and functions. Eliglustat may be used in substrate reduction therapy in diseases in which the enzyme glucocerebrosidase (GCase), responsible for the breakdown of glucocerebroside, is deficient.

FDA_UNII_Code

DR40J4WA67

FULL_SYN

N-((1R,2R)-2-(2,3-Dihydro-1,4-benzodioxin-6-yl)-2-hydroxy-1-(pyrrolidin-1-ylmethyl)ethyl)octanamide

ELIGLUSTAT

Eliglustat

GENZ 99067

GENZ-99067

Has_Salt_Form

http://ncicb.nci.nih.gov/xml/owl/EVS/Thesaurus.owl#C96283

Has_Target

http://ncicb.nci.nih.gov/xml/owl/EVS/Thesaurus.owl#C127919

label

Eliglustat

NCI_Drug_Dictionary_ID

805824

Preferred_Name

Eliglustat

prefixIRI

Thesaurus:C96282

Semantic_Type

Organic Chemical

Pharmacologic Substance

UMLS_CUI

C3272698

subClassOf

http://ncicb.nci.nih.gov/xml/owl/EVS/Thesaurus.owl#C471

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